CClinicalTrials.gg
RecruitingNCT04157049Updated Aug 19, 2026

Alpha-1 Research Registry

An observational study in Alpha 1-Antitrypsin Deficiency, sponsored by Alpha-1 Foundation. Recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2026-08-19.

Sponsored by Alpha-1 Foundation · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
5,000
Sex
All
01

Study summary

The Alpha-1 Research Registry is a confidential database made up of individuals diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1) and individuals identified as Alpha-1 carriers. The Registry was established to facilitate research initiatives and promote the development of improved treatments and a cure for Alpha-1.

Read the detailed description

The purpose and goal of the Alpha-1 Research Registry (Registry, or group of patients) will be to obtain uniform, longitudinal (over the course of time), complete and accurate data that can be organized, and made available for the public to query. The collective number of Registry members enables investigators to enroll sufficient subjects to carry out their studies. The community benefits from having more research and potential therapies performed in their disease. Regular updates from patients will give objective data-points to measure the progression of disease.

02

Conditions studied

  • Alpha 1-Antitrypsin Deficiency

Keywords

  • AATD
  • ALPHA 1
  • COPD
  • AAT
  • ZZ
  • MZ
  • CARRIER
  • MM
  • MS
  • PiZZ
03

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

The current protocol seeks to enroll 4,000 individuals of all age, race, and sex.

Inclusion criteria

  • Patients diagnosed with Alpha-1 Antitrypsin Deficiency (PiZZ, PiZNull, PiSZ etc.)
  • Alpha-1 carriers (PiMZ, PiMS etc.)

Exclusion criteria

Exclusion Criteria:

  • Failure to provide informed consent
  • Normal healthy individuals (MM)
04

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
5,000 participants (estimated)
Target follow-up
3 Years
Patient registry
Yes

Groups and cohorts

  • Alpha-1 Diagnosed Individuals

    Larger patient cohorts are needed to support the clinical trials coming in the next 3-5 years. Despite widespread invitations to the Alpha-1 community from the Alpha-1 Foundation Research Registry, it is estimated that the Alpha-1 Foundation Research Registry now contains \<40% of the identified PiZZ individuals in the US.

  • Carriers of Alpha-1

    Larger patient cohorts are needed to support the clinical trials coming in the next 3-5 years. Despite widespread invitations to the Alpha-1 community from the Alpha-1 Foundation Research Registry, it is estimated that the Alpha-1 Foundation Research Registry now contains \<40% of the identified PiZZ individuals in the US.

05

What researchers measure

Primary outcomes

  1. Establish the Alpha-1 Research Registry using REDCap

    To gather accurate patient data for longitudinal prospective follow up/analysis of Alpha-1 progression.

    Time frame: 8 years

06

Study locations

1 of 1 sites recruiting
  • Alpha-1 Foundation
    Coral Gables, Florida 33134, United States
    • Alison Keaveny, MBBS · Contact · akeaveny@alpha1.org · 1-877-228-7321
    • Nadine Nuchovich, MPH · Contact · nnuchovich@alpha1.org · 1-877-228-7321
    • Jeanine D'Armiento, MD, PhD · Principal investigator
    Recruiting
07

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT04157049
Lead sponsor
Alpha-1 Foundation
Responsible party
Sponsor
First posted
Nov 8, 2019
Start date
Jun 20, 2019
Primary completion
Jun 20, 2029 (estimated)
Completion
Jun 20, 2029 (estimated)
Last update
Aug 19, 2026

Study contacts

Alison Keaveny, MBBS
Contact
akeaveny@alpha1.org
1-877-228-7321 ext. 252
Randel Plant
Contact
rplant@alpha1.org
1-877-228-7321 ext. 306 or 245
Jeanine D'Armiento, MD, PhD
principal investigator · Alpha-1 Foundation

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Interested in this study?

Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.

Contact study team

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Start the discussion